Current therapy of primary systemic (AL) amyloidosis with oral melphalan and prednisone remains unsatisfactory, with a median survival of only 13 months. Between 1996 and 2003, 93 patients with biopsy-proven AL amyloidosis were enrolled in a prospective US national cooperative group trial. Treatment schema consisted of induction therapy with pulse dexamethasone (DEX), followed by maintenance th...

RATIONALE Apolipoprotein A-1 (ApoA-1)-related amyloidosis is characterized by the deposition of ApoA-1 in various organs and can be either hereditary or nonhereditary. It is rare and easily misdiagnosed. Renal involvement is common in hereditary ApoA-1 amyloidosis, but rare in the nonhereditary form. PATIENT CONCERNS We reported two cases with ApoA-1 amyloidosis, a 64-year-old man suffering f...

RATIONALE Hereditary amyloidosis is diagnosed worldwidely with an increasing incidence. As the most common form, transthyretin-related hereditary amyloidosis (ATTR amyloidosis) is an autosomal dominant inherited disease due to mutations of TTR. Over the past several decades, more than 130 mutations have been reported. Previous studies suggested that ATTR amyloidosis initially showed polyneuropa...

SEE PAGE 126 C ardiac amyloidosis (CA) is part of a systemic disease characterized by the deposition of amyloid in multiple tissues. Although the heart is often the predominant organ involved in systemic amyloidosis, this is not always the case, as CA of varying severity may be present when a patient with amyloidosis presents with symptoms related to another organ. Thus, in familial amyloidosis...

Extensive cardiac amyloid deposition in systemic AL amyloidosis is associated with a grave prognosis. Heart transplantation is rarely performed because of the systemic and progressive nature of the disease. Patients with severe cardiac amyloid infiltration are ineligible for the preferred treatment of melphalan chemotherapy with stem cell transplantation (SCT) rescue because of the high risk fo...

For example, it has been found that the serum protein patterns of patients suffering from amyloidosis are often abnormal (1-4), that abnormal serum globulins and amyloid may both be present in patients with multiple myeloma (5-7), and that the incidence of amyloidosis is high in hyperlmmunized horses (8). Yet no single abnormality of the blood proteins has thus far been clearly related to amylo...

Transthyretin is primarily synthesized in the liver and transports thyroxine vitamin A body. The transthyretin when dissociated into monomers can misfold ultimately form amyloid fibrils. There are two types of ATTR amyloidosis: hereditary (caused by mutations TTR gene) wild-type (also referred to as senile systemic amyloidosis). Amyloid cardiomyopathy develop patients with both amyloidosis, has...